Gene Trouble: 9-month-old KJ Muldoon was diagnosed with a rare genetic condition, affecting just 1 in 1.3M babies. Faced with a grim prognosis (half of babies with the condition die within a week), doctors developed a customized gene-editing treatment that could spot and fix a single DNA letter among 3 billion in the human genome. Three infusions later, the infant is now reportedly meeting developmental milestones — potentially offering a blueprint to treat similar rare genetic conditions.
https://www.nytimes.com/2025/05/15/health/gene-editing-personalized-rare-disorders.html